Financial Performance - Revuforj net revenue in 2Q25 reached $28.6 million, representing a 43% quarter-over-quarter growth[6] - Niktimvo net revenue reported by Incyte in 2Q25 was $36.2 million, a substantial increase from $13.6 million in the first two months of Q1 launch[9] - Syndax reported $9.4 million in collaboration revenue from Niktimvo in 2Q25, achieving profitability in the first full quarter[9] - Syndax holds a strong financial position with $518 million in cash and equivalents as of June 30, 2025[28] Product Pipeline and Market Opportunity - Syndax estimates a $5B+ Total Addressable Market (TAM) for Revuforj and another $5B+ TAM for Niktimvo, indicating a $10B+ market opportunity across R/R and frontline indications[4, 30] - The initial Niktimvo indication represents a $2B U S market opportunity, with potential for label and geographic expansion leading to a $5B+ TAM[19, 20] - Approximately 33% of KMT2A patients are estimated to have proceeded to hematopoietic stem cell transplantation (HSCT) following Revuforj treatment[6, 12] Clinical Data and Development - Phase 2 Relapsed/Refractory (R/R) mNPM1 AML data for Revumenib showed a 26% (20/77) CR+CRh rate and a 48% (37/77) Overall Response Rate (ORR)[26] - Phase 1 R/R NUP98r AML data for Revumenib showed a 60% (3/5) ORR[26] - Over 80% of bone marrow transplant centers have ordered Niktimvo[18]

Financial Performance - Revuforj generated $20.0 million in net revenue in 1Q25[3] - Niktimvo generated $13.6 million in net revenue in 1Q25 (reported by Incyte)[3] - The company held $602.1 million in cash and cash equivalents as of March 31, 2025[3] - Total revenue for 1Q25 was $20.0 million[24] - Net loss for 1Q25 was $84.8 million[24] Product Development and Milestones - Syndax initiated a pivotal frontline trial of revumenib with ven/aza in mNPM1 and KMT2Ar patients unfit for intensive chemo in 1Q25[3] - The company completed the submission of Revuforj sNDA for R/R mNPM1 AML in April 2025[3] - Syndax anticipates reporting Phase 1 data from a frontline trial evaluating revumenib with I C (7+3) in 4Q25[27] - Over 1,250 Niktimvo infusions have been ordered YTD[18] Market Access and Adoption - Two-thirds of accounts ordering Revuforj have ordered multiple times[10] - Approximately 95% of all Bone Marrow Transplants (BMTs) ordered are from top accounts[18]

Financial Data and Key Metrics Changes - For Q4 2024, Syndax Pharmaceuticals reported $7.7 million in net revenue from Revuforj, marking the company's first quarter with product revenue [10][46] - Operating expenses for Q4 were $104 million, which included $65.5 million in research and development expenses and $37.7 million in selling, general, and administrative expenses [46] - The company maintained a strong financial position with $692.4 million in cash, equivalents, and investments at year-end [47] Business Line Data and Key Metrics Changes - Revuforj, the first-in-class menin inhibitor, launched in November 2024, has shown strong early demand with $7.7 million in net revenue from the first five weeks of sales [10][11] - Niktimvo, launched in January 2025 for chronic graft versus host disease, is expected to capture a significant portion of the $1.5 billion to $2 billion market for third-line or later chronic GVHD treatment in the US [32] Market Data and Key Metrics Changes - Approximately 33% of Tier 1 and Tier 2 accounts have ordered Revuforj, indicating strong early penetration in key treatment centers [21][22] - The company is seeing rapid inclusion of Revuforj in payer coverage, with 56% of commercially covered lives and 53% of all managed care lives having formal coverage policies in place [25] Company Strategy and Development Direction - The company aims to expand Revuforj's indication to include mutant NPM1 AML by filing a supplemental new drug application in the upcoming quarter [12] - Syndax is focused on building a robust pipeline in oncology, with plans to extend its products into earlier line indications and develop additional targeted therapies [99][100] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position for long-term growth, highlighting the strong market opportunities with Revuforj and Niktimvo [54] - The management team emphasized the importance of building a habit among physicians for prescribing Revuforj, which is expected to become the standard of care for KMT2A translocations [24][60] Other Important Information - The company received $350 million upfront from Royalty Pharma in exchange for a capped royalty on US net sales of Niktimvo, indicating the product's multi-billion-dollar potential [15] - The company expects R&D expenses for Q1 2025 to be between $65 million and $70 million, with total R&D plus SG&A expenses projected to be between $105 million and $110 million [48] Q&A Session Summary Question: Inventory levels and repeat prescribers for Revuforj - Management indicated that inventory levels are expected to remain stable at about two to three weeks, with revenue growth potentially increasing absolute inventory levels [62] - Approximately one-third of Tier 1 and Tier 2 accounts have ordered Revuforj, with most accounts having prescribed it more than once, indicating positive early adoption [65] Question: Activation of Tier 1/2 accounts and comparison to historic AML launches - Management expects continued activation of Tier 1 and Tier 2 accounts over the next 10 months, with a focus on the top 200 treatment centers [73] - The unique setup of Revuforj, including diagnostic testing for KMT2A patients, is expected to drive its adoption compared to other targeted therapies [78] Question: Off-label use and Expanded Access Program (EAP) - Anecdotal evidence suggests some off-label use of Revuforj, particularly in combination treatments, but specific numbers are not yet available [86] - The EAP remains lightly used, with no additional patients expected to transition from it at this time [87] Question: Future company vision and pipeline expansion - The company aims to build a larger pipeline in oncology and expects to have multiple commercial products by 2030, focusing on targeted therapies [100][101] Question: Safety feedback and physician experience with Revuforj - Feedback on Revuforj's safety profile has been positive, with comprehensive support provided to physicians through clinical nurse educators [110] Question: Gross to net adjustments and patient assistance programs - The gross to net adjustments for Revuforj are expected to remain tight due to the limited distribution channel, with a patient assistance program available for uninsured or underinsured patients [118][120] Question: Transplant dynamics and patient treatment pathways - Physicians are interested in getting patients to transplant, particularly for KMT2A, with expectations that many patients will return to maintenance therapy post-transplant [122]